Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia

Completed · Phase 2

Conditions studied: Severe Thalassemia

In brief

This study is being done to determine if blood cell transplants, with either bone marrow or cord blood from unrelated donors, are effective in children with severe thalassemia and if this treatment approach has acceptable risks and side effects. This study includes a preparative regimen with Hydroxyurea, Alemtuzumab, Fludarabine, Thiotepa and Melphalan that provides intense host immunosuppression without myeloablation. The primary hypothesis is that this regimen will promote stable engraftment of unrelated donor hematopoietic cells, support normal erythropoiesis, and result in an event free survival of \> 75% of children with thalassemia major.

Key facts

Study ID

NCT01005576

Run by

Washington University School of Medicine

People needed

21

Starts

2010-01-01

Expected to finish

2014-07-01

Last updated by the study team

2017-12-13

Who can join

Age: 1 and older, up to 16. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 1-16.00 years old
• Have transfusion dependent thalassemia major
• Shall not have an HLA-matched family donor
• Must have a suitably matched unrelated marrow donor or UCB product
• Lansky score >/= 70
• Adequate pulmonary, renal, liver, and other organ function as defined in protocol
• Negative pregnancy test
• Adequate total nucleated cell or CD34+ dose of product as defined in protocol
• Iron chelation must be discontinued >/= 48 hours prior to conditioning regimen

You may not qualify if…

• Pregnant or breastfeeding
• HIV positive
• Prior allogeneic marrow or stem cell transplantation

Where it is running

• Regents of University of California- UCLA — Los Angeles, California, United States
• Children's Hospital and Research Center at Oakland — Oakland, California, United States
• Children's National Medical Center — Washington D.C., District of Columbia, United States
• University of Miami — Miami, Florida, United States
• All Children's Research Institute, Inc. — St. Petersburg, Florida, United States
• Emory University — Atlanta, Georgia, United States
• Children's Memorial Hospital — Chicago, Illinois, United States
• Dana Farber Cancer Institute — Boston, Massachusetts, United States
• University of Michigan — Ann Arbor, Michigan, United States
• Washington University — St Louis, Missouri, United States
• The University of North Carolina at Chapel Hill — Chapel Hill, North Carolina, United States
• Duke University — Durham, North Carolina, United States
• The Research Institute at Nationwide Children's Hospital — Columbus, Ohio, United States
• Oregon Health and Science University — Portland, Oregon, United States
• The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Vanderbilt University — Nashville, Tennessee, United States
• UT Southwestern Medical Center — Dallas, Texas, United States
• Methodist Healthcare System of San Antonio — San Antonio, Texas, United States

Full record on ClinicalTrials.gov

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